In a lab room, a toddler, deaf from birth, sits while a tone plays. There’s no reaction. His face does not change. Six weeks ...

A first-of-its-kind gene therapy that can restore hearing in children born deaf has just cleared its final hurdle—and, in a ...

Regeneron is seeing double-digit revenue growth and has more than 50 therapies in its pipeline, making it a good long-term ...

Danish dermatology specialist Leo Pharma is paying $50 million for U.S. gene writing biotech Replay and its preclinical gene ...

An experimental gene therapy for a rare form of genetic deafness has successfully restored hearing in children and adults, ...

Astellas Pharma has done some spring cleaning of its pipeline, dropping two phase 1 candidates and slamming the brakes on a ...

Travis used to sleep through everything. Then his mother laughed in the car and woke him up, the first time he had ever heard ...

Ray Therapeutics has received priority medicines (PRIME) designation from the European Medicines Agency (EMA) for its RTx-015 ...

Regeneron’s drug restored hearing in 11 of 12 children in a rare, inherited condition that causes deafness.

Cell and gene therapies have a huge curative potential but come with a large price tag that can result in access barriers. At ...

News of the submission, which would be the first for UniQure’s closely watched treatment, comes as the therapy faces an ...

Cell and gene therapies are currently niche products, even within specialty pharmacy, drugs that, by most definitions, are ...